Survey of a treatment for congenital thrombotic thrombocytopenic purpura
Official title: Special Drug Use Surveillance of ADZYNMA Intravenous 1500 (All-Case Investigation)
Run by Takeda · NCT06441578 · Data updated 2026-11-12
This study is testing a new treatment called recombinant ADAMTS13 to see if it helps people with congenital thrombotic thrombocytopenic purpura feel better and stay healthy over 18 months.
Quick facts
| Study type | Observational |
|---|---|
| Sex | All |
| Enrollment | 40 (estimated) |
| Sponsor | Takeda Industry-sponsored |
| Locations | 1 site (Tokyo, Tokyo) |
| Status | Recruiting |
| Started | May 30, 2024 |
| Main results expected | September 30, 2032 |
| Study ends | September 30, 2032 |
| Registry record updated | October 13, 2026 |
| Trial ID | NCT06441578 on ClinicalTrials.gov |
Is this trial a fit for you?
✓Likely a good fit
Ideal candidates for this study are individuals diagnosed with congenital thrombotic thrombocytopenic purpura who are being treated with recombinant ADAMTS13.
✕Probably not a fit
Patients without congenital thrombotic thrombocytopenic purpura or those not receiving recombinant ADAMTS13 will not benefit from this study.
A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.
What this trial studies
This observational study surveys the use of recombinant ADAMTS13 in participants with congenital thrombotic thrombocytopenic purpura (cTTP) in Japan. Participants will receive intravenous injections of recombinant ADAMTS13 according to their clinic's standard practice, while the study will monitor for any side effects and assess the treatment's effectiveness in improving or preventing cTTP over an 18-month period. The study aims to gather data on the safety and efficacy of this treatment without direct involvement from the sponsor in patient care.
Why it matters
Potential benefit: If successful, this study could provide valuable insights into the safety and effectiveness of recombinant ADAMTS13 for patients with cTTP.
How similar studies have gone: While this approach is observational, similar studies on recombinant ADAMTS13 have shown promise in treating TTP, indicating potential for success.
Who can join: the full rules
Show full inclusion / exclusion criteria
Inclusion Criteria: \- All participants with congenital thrombotic thrombocytopenic purpura (cTTP), treated with recombinant ADAMTS13 Exclusion Criteria: \- None
Where it's running
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Takeda Selected Site
Tokyo, Tokyo, Japan Recruiting
Study contacts
- Study coordinator: Takeda Contact
- Email: medinfoUS@takeda.com
- Phone: +1-877-825-3327
Similar studies
Other studies for the same condition or treatment. Recruiting studies are listed first.
-
cTTP patients treated with recombinant ADAMTS13: what happened after the early access program
This study looks at people with congenital TTP who received recombinant ADAMTS13 to see how treatment changes their health before and after use.
-
Adzynma in people with congenital TTP: a look at safety and antibody risk
This study checks whether people with congenital TTP who have already received Adzynma develop neutralizing antibodies against Adzynma within six months of starting treatment.
-
Long-term outcomes for people with immune TTP in the French national TMA cohort
This project uses past medical records to see long-term health outcomes and complications in people in France who had immune thrombotic thrombocytopenic purpura (iTTP).
-
Understanding the immune response in Thrombotic Thrombocytopenic Purpura
This study is trying to understand how certain immune cells behave in people with Thrombotic Thrombocytopenic Purpura to see if they can help predict relapses and improve treatment.
-
Registry for patients with immune-mediated thrombotic thrombocytopenic purpura in Italy
This study is tracking patients with immune-mediated thrombotic thrombocytopenic purpura in Italy to see how the disease affects them over time and what their treatment outcomes are.
-
Registry for patients with Thrombotic Thrombocytopenic Purpura in Turkey
This study is collecting information from patients with Thrombotic Thrombocytopenic Purpura in Turkey to see how the disease is treated and how it affects their health over time.
How to take part
- Review the eligibility criteria above with your treating physician.
- Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
- Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.